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Translational Therapy in Patients With Osteogenesis Imperfecta - A Pilot Trial on Treatment With the Rankl-Antibody Denosumab (OI-AK)

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ClinicalTrials.gov Identifier: NCT01799798
Recruitment Status : Completed
First Posted : February 27, 2013
Last Update Posted : January 27, 2015
Sponsor:
Information provided by (Responsible Party):
Dr. med. Joerg Oliver Semler, University of Cologne

February 14, 2013
February 27, 2013
January 27, 2015
February 2013
January 2015   (Final data collection date for primary outcome measure)
Changes of bone mineral density (BMD [g/cm2]) in lumbar spine after 36 weeks of treatment with denosumab. Changes will be calculated between baseline and study week 48. [ Time Frame: 48 weeks ]
Same as current
Complete list of historical versions of study NCT01799798 on ClinicalTrials.gov Archive Site
  • Decrease of osteoclastic activity measured by urinary deoxypyridinoline (DPD). [ Time Frame: 14 days (DPD) ]
  • Parathormone in study week 12, 24, 36 and 48 compared to baseline. [ Time Frame: 12 weeks ]
    Descriptive statistical analysis
  • N-Telopeptides in study week 12, 24, 36 and 48 compared to baseline. [ Time Frame: 12 weeks ]
    descriptive statistical analysis
  • Osteocalcin in study week 12, 24, 36 and 48. [ Time Frame: 12 weeks ]
    descriptive statistical analysis
Same as current
Not Provided
Not Provided
 
Translational Therapy in Patients With Osteogenesis Imperfecta - A Pilot Trial on Treatment With the Rankl-Antibody Denosumab
TRANSLATIONAL THERAPY IN PATIENTS WITH OSTEOGENESIS IMPERFECTA - A PILOT TRIAL ON TREATMENT WITH THE RANKL-ANTIBODY DENOSUMAB

Pilot study to assess the efficacy of a therapy with the RANKL-antibody denosumab in children 5-10 years of age with mutation in COL1A1 or COL1A2 leading to Osteogenesis imperfecta. Efficacy will be assessed by DXA measurements at the lumbar spine of the areal bone mineral density (BMD) which is the most frequently used parameter in trials investigating osteoporosis.

The hypothesis of the study is:

Osteoclastic activity which is increased in OI could be reduced by inhibition of osteoclast maturation. Denosumab inhibits maturation of the osteoclasts by inhibiting RANKL. BMD could be increased during a 36 week treatment course with denosumab measured after 48 weeks.

Not Provided
Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Osteogenesis Imperfecta
Drug: Denosumab
Denosumab will be given subcutaneously in a dosage of 1mg/kg body weight every 12 weeks. 4 interventions are planned until trial week 36. There is no control group planned.
Experimental: Denosumab subcutaneously
Intervention: Drug: Denosumab
Semler O, Netzer C, Hoyer-Kuhn H, Becker J, Eysel P, Schoenau E. First use of the RANKL antibody denosumab in osteogenesis imperfecta type VI. J Musculoskelet Neuronal Interact. 2012 Sep;12(3):183-8.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
10
January 2015
January 2015   (Final data collection date for primary outcome measure)

Inclusion Criteria

  • Male or female subjects between 5 years and 10 years of age with molecular proven Osteogenesis imperfecta (COL1A1/A2 mutation)
  • Subjects must have been treated for a minimum of 2 years with bisphosphonates prior to study entry

Exclusion Criteria:

  • Hypocalcemia (<1.03 mmol/l ionized Calcium)
  • Subjects with reduced renal function (estimated GFR (Schwartz formula) <30ml/min/1.73m2)
  • Any other abnormal finding such as physical examination or laboratory evaluation, in the opinion of the investigator that is indicative of a disease that would compromise the safety of the patient when getting denosumab s.c.
Sexes Eligible for Study: All
5 Years to 11 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Germany
 
 
NCT01799798
Uni-Koeln-1574
No
Not Provided
Not Provided
Dr. med. Joerg Oliver Semler, University of Cologne
University of Cologne
Not Provided
Principal Investigator: Joerg Oliver Semler, MD University Cologne, Childrens Hospital, Cologne, Germany
University of Cologne
January 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP